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American Association for the Advancement of Science, Science Translational Medicine, 165(4), 2012

DOI: 10.1126/scitranslmed.3004108

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Genetic Correction of Human Induced Pluripotent Stem Cells from Patients with Spinal Muscular Atrophy

This paper is available in a repository.
This paper is available in a repository.

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Data provided by SHERPA/RoMEO

Abstract

Motor neurons generated from genetically corrected iPSCs derived from patients with spinal muscular atrophy show rescue of the disease phenotype.